A Long-Term Follow-Up Safety Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder
研究概览
简要总结
The goal of this observational study is to assess the safety and tolerability of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).
详细描述
Subjects who received AVR-RD-02 in the preceding treatment study (NCT04145037) and who meet all eligibility criteria may participate. Subjects will be asked to return for study visits annually for 14 years to assess measures of safety and persistence of AVR-RD-02 treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Subjects who received AVR-RD-02
Subjects who received AVR-RD-02 in a separate parent trial
干预措施: AVR-RD-02 (Biological)
结局指标
主要结局
Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder
时间窗: Baseline to Year 14
Change from baseline over time in hemoglobin concentration
时间窗: Baseline to Year 14
Change from baseline over time in platelet count
时间窗: Baseline to Year 14
次要结局
- Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood(Baseline to Year 14)
- Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis(Baseline to Year 14)
- Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood(Baseline to Year 14)
- Presence of replication competent lentivirus (RCL)(Baseline to Year 14)
- Change from baseline over time in anti-GCase total antibodies and subsequent titers(Baseline to Year 14)
研究者
John Bernat
Clinical Associate Professor
University of Iowa